Prospective, Open-label, Single-arm, Multicentre Phase 3 Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases

Octapharma1 site in 1 country50 target enrollmentAugust 4, 2021

ConditionsPrimary Immune Deficiency

InterventionsNewnorm

DrugsNewnorm

Overview

Phase: Phase 3
Intervention: Newnorm
Conditions: Primary Immune Deficiency
Sponsor: Octapharma
Enrollment: 50
Locations: 1
Primary Endpoint: Rate of Serious Bacterial Infections
Status: Active, not recruiting
Last Updated: 7 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases

Registry

clinicaltrials.gov

Start Date

August 4, 2021

End Date

September 1, 2026

Last Updated

7 months ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Octapharma

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Age of ≥2 years and ≤75 years
•Documented and confirmed diagnosis of PID as defined by European Society of Immunodeficiencies (ESID) and the Pan American Group for Immune Deficiency (PAGID) and requiring immunoglobulin replacement therapy due to hypogammaglobulinaemia or agammaglobulinaemia. The exact type of PID must be recorded.
•At least 12 weeks of regular treatment before the screening visit (i.e., with a stable dosing interval) with any IVIG, SCIG, or fSCIG, with a stable IgG dose between 200 and 800 mg/kg/month. A stable dose is defined as one that deviates less than ±25% from the mean dose for all infusions within this 12-week period before screening.
•Trough level of IgG ≥5 g/L at screening and documentation of an IgG trough level of ≥5 g/L at least once within the previous 12 weeks.
•Freely given written informed consent from adult patients or freely given written informed consent from the patient's parent(s)/legal guardian(s) and written informed assent from paediatric or adolescent patients in accordance with the applicable regulatory requirements, before any study-specific procedure takes place.
•Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.

Exclusion Criteria

•Any acute infection requiring IV antibiotic treatment within 2 weeks before the screening visit or during the screening period, or any SBI within the 3 months prior to the screening visit or during the screening period.
•The patient has isolated specific antibody deficiency disorder, isolated IgG subclass deficiency, or transient hypogammaglobulinaemia of infancy.
•Current medical condition or history of condition known to cause secondary immune deficiency, for example, chronic lymphocytic leukaemia, lymphoma, multiple myeloma, or chronic or recurrent neutropenia (absolute neutrophil count \<1000/μL).
•Known history of ADRs to IgA contained in other products.
•Body mass index \>40 kg/m
•Exposure to blood or any blood product or plasma derivative other than IgG for PID within 3 months before the first infusion of Newnorm.
•History of or ongoing severe hypersensitivity, e.g., anaphylaxis or severe systemic response, or persistent reactions to blood or plasma-derived products, or to any component of Newnorm (such as glycine).
•Severe liver dysfunction (alanine aminotransferase \[ALT\] \>3 times the upper limit of normal for the expected normal range for the testing laboratory) at screening.
•Known protein-losing enteropathies or proteinuria (known urinary protein loss of \>1 g/24 h, or dipstick proteinuria of ≥3+).
•Moderate to severe renal dysfunction (per investigator discretion based on estimated glomerular filtration rate \[eGFR\] ≤44 mL/min/1.73 m2, as defined by KDIGO Clinical Practice Guideline) or predisposition to acute renal failure (e.g., any degree of pre-existing renal dysfunction in presence of additional acute renal failure risk factors, e.g. routine treatment with known nephrotoxic drugs).

Arms & Interventions

Newnorm

Newnorm is a 20% human normal immunoglobulin for SC infusion

Intervention: Newnorm

Outcomes

Primary Outcomes

Rate of Serious Bacterial Infections

Time Frame: 52 weeks

Rate of SBIs (defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess) per person-year on treatment.

Average total IgG concentration

Time Frame: 16 weeks

Average total IgG concentration (Cav) on steadystate dosing.

Secondary Outcomes

Hospitalisations due to infection(52 weeks)
Infections of any type of seriousness(52 weeks)
Time to resolution of infections(52 weeks)
Episodes of fever(52 weeks)
Child Health Questionnaire-Parent Form(52 weeks)
SF-36 Health Survey(52 weeks)
Non-compartmental PK analyses of all available PK profiles (IVIG and SCIG)(16 weeks)
IgG Levels(16 weeks)
Days lost from work, school, kindergarten, or day care due to infection(52 weeks)
Use of antibiotics(52 weeks)